Timeline


The journey from research to potential treatment.

How development progresses

The development of gene therapy follows a structured path, moving from early research to clinical application. Each stage builds on the previous one and must meet strict scientific and regulatory standards.

GAND syndrome timeline chart from 2025 to 2028, showing various research phases including development, testing, and regulatory steps for different projects, with labels for planning, study, protocol planning, and a highlighted green section.

The process begins with fundamental research and understanding of the gene and disease mechanism. This is followed by preclinical development, where potential therapies are tested in controlled environments.

If successful, the therapy moves into clinical trials, where safety and effectiveness are evaluated in patients. Only after passing these stages can a treatment become widely available.

Key development phases

Girl with GATAD2B syndrome (GAND)

A long but meaningful journey

Developing a gene therapy can take several years. While the timeline may seem long, each step is essential to ensure safety, effectiveness, and long-term impact.

Every milestone reached represents real progress toward a future treatment.